Freeline Therapeutics Holdings plc Reports Positive Initial Clinical Data from First Cohort of Phase 1/2 Galileo-1 Trial of FLT201, Its Novel Gene Therapy Candidate, in Gaucher Disease
No elevations in liver transaminase levels during the same time periods. Alanine-transaminase (ALT) and aspartate-transaminase (AST) levels remained in the normal range in both patients. Robust increases in plasma GCase levels. Patient 1 showed a nearly 700-fold increase over baseline to more than 70 µmol/L/h as of 12 weeks post-dosing. Patient 2 showed a similarly robust response, with a greater than 300-fold increase over baseline to approximately 30 µmol/L/h as of four weeks post-dosing. Normal plasma GCase levels range from 0.3 to 1.2 µmol/L/h (mean: 0.58 µmol/L/h). Normalization of leukocyte GCase activity, demonstrating cellular uptake of GCase from the plasma. Leukocyte GCase activity reached normal levels in patient 1 within four weeks of dosing and remained normal as of the last measurement. Similarly, leukocyte GCase activity in patient 2 reached normal levels within four weeks of dosing. Leukocytes are validated markers for broad cellular uptake in Gaucher disease. Both patients had normal hemoglobin levels at baseline and have remained in the normal range at each weekly assessment, including those taken after coming off enzyme replacement therapy or substrate replacement therapy. Given the compelling safety profile and robust enzyme activity at the 4.5x1011 vg/kg dose, a third patient has been scheduled for dosing in this first cohort to gather additional data before deciding whether to continue at the current dose or explore a higher dose. Three additional study patients have been identified and are in the process of being scheduled for dosing.